About the Report
Gene therapies like SKYSONA offer a beacon of hope for patients with devastating disorders, but they also carry significant risks and unanswered questions. This report provides an unvarnished look at the successes, limitations, and areas for improvement in this rapidly evolving field. Inside, you’ll find:
- A deep dive into SKYSONA’s approval timeline and regulatory challenges.
- Detailed safety data, including genetic mutations and outcomes in affected patients.
- Insights into Bluebird bio’s financial and operational strategies in a turbulent market.
- A roadmap for addressing long-term safety concerns in gene therapy development.
Equip yourself with the knowledge needed to navigate the complex world of gene therapy for neurological diseases.
Beacon Gene Therapy
How the most complete gene therapy database can help you
What we cover
We included trial and drug records for preclinical, active, approved, and discontinued:
- RNA/DNA Viral vector-based therapies
- DNA Non-viral vector-based therapies
- Genetically Modified cell therapies
- Empty Vector Trials
- Other comparable modalities within gene therapy, whether cell-free or cell-based
- Beacon Gene Therapy covers all genetically driven non-oncology indications that utilize gene therapy to mediate the condition.
How Beacon Gene Therapy works
Search the clinical trial and drug landscape by gene/mutation, editing technology (CRISPR/Cas9, Cas-CLOVER, etc.), gene delivery system, promoter, etc., instantaneously and/or extract the data points you need to conduct more complex analysis.
Our unique Milestones filter and visualization highlights past, present, and future drug development milestones including drug and trial readouts, asset history, and regulatory announcements. This enhancement provides an accurate, timely and exhaustive single-drug timeline allowing you to benchmark progress in the gene therapy space.
If publicly available, information regarding immunogenicity is included (unique to Beacon Gene Therapy).
Commercial intelligence data expansion: Gain unparalleled access to in-depth company profiles and the latest commercial deals. Monitor your competitors and opportunities as well as benchmark deals and evaluate potential partners. Learn more by downloading the factsheet.
You May Also Be Interested In...
Strategic Insights for Biopharma Innovation Report
H1 2024 Gene Therapy Landscape Review
Investment Trends in Early-Stage Biopharma: A Beacon Data Perspective
Find Out More
Beacon is the essential decision-support tool for developers of complex therapeutics. Our market-defining, proprietary ontologies, combined with the most accurate and comprehensive life sciences data, provide our customers with unparalleled visibility of the drug and trial landscape.
With Beacon, you can make drug development decisions with confidence. This is why 23 of the world’s top 25 drug developers trust us.
At Beacon, we are committed to empowering our pharmaceutical and biotech industry partners with accurate, comprehensive data. We are not just a tool; we see ourselves as a partner in the drug development journey. Contact us to learn how we help you.
